Funding the Fight Against Duchenne Muscular Dystrophy

Funding the Fight Against Duchenne Muscular Dystrophy

From patient to research money man. He suffers from Duchenne Muscular Dystrophy and is now funding scientists on the brink of a breakthrough.

Duchenne Muscular Dystrophy, DMD, is an inherited disorder that involves rapidly worsening muscle weakness. It is one of nine types of muscular dystrophy. Men are more likely than women to develop symptoms of the disease; approximately one out of every 3,600 male infants develops the disease.

CAUSES: It is caused by a defective gene for dystrophin, a protein in the muscles.

SYMPTOMS: Symptoms of DMD can include fatigue, mental retardation, muscle weakness that begins in the legs and pelvis area causing frequent falls. Also, progressive difficulty walking is a sign of DMD. (www.ncbi.nlm.nih.gov)

COMPLICATIONS: There are a series of complications related to DMD. Complications can include heart disease, congestive heart failure, deformities, heart arrthymias, mental impairment, pneumonia or other respiratory infections and also respiratory failure. (www.ncbi.nlm.nih.gov)

TREATMENT: The two drugs Spironolactone and Lisinopril are used for other forms of heart disease. The combination of the two is being studied for the treatment of DMD due to the disease causing breathing and heart muscles to breakdown and fibrosis or scarring to develop.

For the study, the drugs were tested on mice. One group received both drugs at eight weeks of life; the second group at four weeks and the third group received no treatment. At 20 weeks all mice had a normal amount of blood pumped through the heart from the right and left ventricles with each heartbeat.The treated mice showed less heart muscle tissue damage.

In general, it is suggested that individuals with DMD stay active, such as through physical therapy which can help maintain muscle strength and function. Inactivity can worsen the muscle disease.

If this story or any other Ivanhoe story has impacted your life or prompted you or someone you know to seek or change treatments, please let us know by contacting Marsha Hitchcock at mhitchcock@ivanhoe.com.